WE ARE BREAKING BARRIERS

AveXis is a clinical-stage gene therapy company relentlessly focused on bringing gene therapy out of the lab and into the clinical setting for patients and families devastated by rare and life-threatening neurological genetic diseases.

Our initial product candidate, AVXS-101, is our proprietary gene therapy currently in development for the treatment of spinal muscular atrophy (SMA) Type 1, the leading genetic cause of infant mortality, and SMA Type 2.

The AVXS-101 Design

We’ve developed AVXS-101 to possess four key elements of an optimal gene therapy approach to SMA.

01

Recombinant AAV9 Capsid Shell is a non-replicating adeno-associated virus capsid used to deliver a functional copy of the human SMN gene across the blood-brain barrier to cells without modifying the patient’s existing DNA

02

Human SMN Transgene is a stable, functioning SMN gene that is introduced into the cell’s nucleus

Continuous Promoter activates the transgene and is designed to allow for continuous and sustained SMN expression

01 Recombinant AAV9 Capsid Shell is a non-replicating adeno-associated virus capsid used to deliver a functional copy of the human SMN gene across the blood-brain barrier to cells without modifying the patient’s existing DNA

02 Human SMN Transgene is a stable, functioning SMN gene that is introduced into the cell’s nucleus

ADDITIONAL TYPES OF SMA

We have recently initiated a Phase 1 clinical trial to investigate AVXS-101 for the treatment of SMA Type 2. Data from this study will help inform potential future development plans in other SMA subtypes.

AND OTHER RARE, LIFE-THREATENING NEUROLOGICAL GENETIC DISEASES

We have exclusive worldwide license agreements to develop and commercialize gene therapy using the AAV9 vector to treat two rare neurological monogenic disorders: Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene.

By clicking this link, you will be leaving AveXis.com to go to a site that is not controlled or endorsed by AveXis. AveXis provides links to third-party sites as a convenience. AveXis is not responsible for the content of these sites or any of its further links. Each of these sites has their own privacy policy and terms of use. Thank you for visiting AveXis.com.